Case Studies in Translational Research

Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes

Harnessing cellular therapeutics for type 1 diabetes mellitus: progress, challenges, and the road ahead

Sneha Narasimhan, MD, PhD

Anti-amyloid antibody therapies in Alzheimer’s disease

Anti‑Amyloid Monoclonal Antibodies for the Treatment of Alzheimer’s Disease

Biogen’s Aduhelm controversy as a case study for accelerated approval biomarkers in Alzheimer’s and related diseases

Carl June, MD

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Fidanacogene Elaparvovec for Hemophilia B — A Multiyear Follow-up Study

Peter Lebowitz, MD, PhD

Development of TAR-200: A novel targeted releasing system designed to provide sustained delivery of gemcitabine for patients with bladder cancer

TAR-210 Erdafitinib Intravesical Delivery System in Non–Muscle-Invasive Bladder Cancer With Select FGFRAlterations: Updated First-in-Human Results

Safer non-viral DNA delivery using lipid nanoparticles loaded with endogenous anti-inflammatory lipids

Multi-stage-mixing to control the supramolecular structure of lipid nanoparticles, thereby creating 4 a core-then-shell arrangement that improves performance by orders of magnitude

Targeting DNA-LNPs to Endothelial Cells Improves Expression Magnitude, Duration, and Specificity

Aluminum-Adsorbed Vaccines and Chronic Diseases in Childhood

CHOP - Vaccine Ingredients: Aluminum

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

A Milestone for Gene-Editing Therapies

Monday, November 10

Christina Coughlin, MD, PhD

Individualised neoantigen therapy mRNA-4157 (V940) plus pembrolizumab versus pembrolizumab monotherapy in resected melanoma (KEYNOTE-942): a randomised, phase 2b study

Personalized RNA neoantigen vaccines stimulate T cells in pancreatic cancer

A neoantigen vaccine generates antitumour immunity in renal cell carcinoma